The U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients 1 year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe.
Data with Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa in late-onset Pompe disease and infantile-onset Pompe disease will be featured as platform and poster presentations at the 17th annual WORLDSymposiumTM, to be held February 8-12, 2021.
The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.
San Diego-based Retrophin is buying Orphan Technologies for $90 million in cash up front and up to $427 million in milestone payments.
Shire’s experimental treatment SHP609 to address cognitive impairment in pediatric patients diagnosed with the rare disease known as Hunter syndrome failed a key trial.
Barely a week after announcing it was moving into a new corporate headquarters, Sangamo Therapeutics announced that the company has dosed the first patient in its Phase I/II gene therapy trial.